Translate Bio Provides MRT5005 Program Updates
-- FDA grants Rare Pediatric Disease designation for MRT5005 for treatment of cystic fibrosis –
-- Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date --
“Our priority is always patient safety and well-being, and specifically during this unprecedented public health crisis, we join our health and local authorities, healthcare providers, and patient advocacy groups in ensuring that patients with CF exercise an abundance of caution to prevent exposure to SARS-CoV-2. As more patients with CF and their physicians choose to avoid unnecessary risk of exposure to SARS-CoV-2, we, like many companies across the healthcare and biopharmaceutical industry, anticipate that patient dosing and follow-up visits in our ongoing trial will be affected,” said Dr.
About Rare Pediatric Disease Designation
The FDA grants Rare Pediatric Disease designation for a disease that is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals ages 18 years or younger and fewer than 200,000 people in
MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization. MRT5005 is being developed to treat all patients with CF, regardless of the underlying genetic mutation, including those with limited or no CFTR protein. The FDA has granted MRT5005 Orphan Drug, Fast Track and Rare Pediatric Disease designation.
About the MRT5005 Phase 1/2 Clinical Trial
The randomized, double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 is designed to enroll at least 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization. Percent predicted forced expiratory volume in one second (ppFEV1), which is a well-defined and accepted endpoint measuring lung function, will also be measured at pre-defined timepoints throughout the trial. The Phase 1/2 clinical trial of MRT5005 for the treatment of CF is being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network. In
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to address the underlying cause of CF and benefit patients; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; Translate Bio’s expectations regarding interruptions in patient dosing and follow-up in the ongoing Phase 1/2 clinical trial of MRT5005; Translate Bio’s expectations on when it will provide an interim data readout from the trial; Translate Bio’s plans for future trials of MRT5005 in pediatric CF patients and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forward,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: Translate Bio’s ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the content and timing of decisions made by the FDA, other regulatory authorities and investigational review boards at clinical trial sites, including decisions as it relates to ongoing and planned clinical trials; Translate Bio’s ability to obtain, maintain and enforce necessary patent and other intellectual property protection; the availability of significant cash required to fund operations; competitive factors; the COVID-19 pandemic; general economic and market conditions and other important risk factors set forth under the caption “Risk Factors” in Translate Bio’s Annual Report on Form 10-K for the fiscal year ended
Source: Translate Bio, Inc.